FDA announces steps to modernise drug development and improve efficiency

US FDA commissioner has announced steps to modernise the drug development process to improve efficiency and promote innovation…


The US FDA Commissioner Dr Scott Gottlieb has put out a statement for the modernisation of drug development, to improve the efficiency of the process and to promote the innovation of targeted therapies.

He wrote how by ‘using new technology platforms such as cell and gene therapies and small molecule drugs that target the genomic basis of disease, there are more opportunities to intervene in the underlying mechanisms that cause a disease, and potentially arrest and even reverse its progress.’

In the FDA statement, Dr Gottlieb wrote about current efforts that are aimed at the improvement of each stage of the drug development process, whilst also focusing on generating evidence, both pre-clinical and clinical, to make regulatory decisions more scientific, efficient and ‘modern’.

The statement mentions how the Center for Drug Evaluation and Research has already approved 45 novel drugs and biologics this year, which is close to the total of 46 in 2017 – the most approved in over 20 years.

Dr Gottlieb wrote how the new treatments offer hope to patients for an improved quality of life, and improved chances of surviving life-threatening illnesses.

Among the diseases mentioned are novel treatments for migraines and an RNA-based therapy to treat polyneuropathy caused by hereditary transthyretin-mediated amyloidosis. 

He wrote of continuing the focus on modernising the approach to clinical trials, and making the overall drug development process more efficient and less expensive. He stressed that regulatory standards would still need to be met, and that by ‘using more sophisticated approaches to learn about the safety and efficacy of treatments, we can reduce the barriers to bringing new science forward and make sure more patients can benefit sooner from improved treatments.’

The statement explains how ‘one of the most promising ways to make drug development more efficient – while enabling providers and patients to get better information about how a new medicine works – is by developing the science around innovative approaches to the design of clinical trials’. The commissioner mentioned that these are approaches that can show more quickly and efficiently how a treatment will impact a specific patient population.

Concluding the statement from the FDA, Dr Gottlieb wrote: “today’s guidance documents are part of the FDA’s science-based mission to modernise clinical trials and advance the development of safe and effective drugs and biologics for the American public.”

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